GOLD was “born” in February 2002, when a group of 30 well know names in the field of Lysosomal Storage Diseases met in Cannes, France to discuss the need for an international collaboration on LSDs. The attendees represented basic and clinical research, treatment centres, patient organisations and pharmaceutical companies.
The meeting, held under the aegis of Lysosomal Diseases Australia, was hosted by a Steering Committee comprising Michael Beck (Germany), John Hopwood (Australia), Edwin Kolodny (US), Christine Lavery (UK), Abbey Meyers (US) and William Sly (US). John Hopwood, who was the driving force for the meeting, set out six reasons why it was timely to consider an international collaboration – or, as he said ‘to seize the moment’.
increasing understanding of aetiology of lysosomal storage diseases
growing number of patient registries and genetic information databases
promising/approved diagnostics and treatments
committed cadre of researchers, clinicians, and patient advocates/educators
model programmes for patient education and support
broadening recognition of the value of coordinated action
The meeting addressed the key challenges in the areas of epidemiology, disease progression, diagnosis and treatment and disease education. From the discussion, there was identified the need to:
understand the global incidence and prevalence of the diseases
have a better understanding of the natural history of the diseases
develop defined clinical endpoints and a better appreciation of their heterogeneity and impact on quality of life and to validate surrogate endpoints for evaluating treatments
standardise and legitimise laboratory testing and to link with clinical and diagnostic services
clarify the importance of early diagnosis in bringing patients to therapy whilst there is still a chance for change
develop wide-scale newborn screening, and for international guidelines for carrier identification and patient diagnosis
build a collaboration for patient databanks, including specimen and tissue banks
provide accurate patient and physician education and create greater awareness and dissemination of important clinical management developments
encourage new therapies
expand the patient base for clinical trials and improve the rate of recruitment to trials
increase funding for education, testing, and large scale screening
speak with one voice to regulatory agencies and the public
Since the inaugural meeting in Cannes, GOLD had been incorporated as a company based in the UK, and registered as a charity. Membership has grown steadily and continues to do so.
Click here to find out more about GOLD membership.